Second FDA Approval for Neuren’s Trofinetide

With the launch of trodinetide’s formulation, Neuren has achieved its second FDA approval:

In December 2025, Neuren Pharmaceuticals announced a milestone achievement with the launch of trodinetide’s formulation, partnering with Acadia Pharmaceuticals to gain U.S. FDA approval for DAYBUX STIX, a novel dye- and preservative-free powder formulation of trofinetide for oral solutions. The formulation of drug trofinetide, as marked under the brand name DAYBUE.

The SAYBUE STIX is a powder formulation for oral solutions to provide great flexibility for patients and caregivers, as it can be mixed with different water-based liquids to modify taste and volume. Original DAYBUE oral solution was the first-ever FDA-approved treatment for Rett syndrome in the U.S., which was approved in March 2023.

CEO John Pilcher’s Statement on Second FDA Approval for Trofinetide:

John Pilcher, CEO of Neuren, has stated that this approval reflects Acadia’s continuous investments in broadening access to the therapy. According to John Pilcher, the neuro team is the most excited team for this approval, as new treatment solutions for Rett syndrome families and constant investments and innovations for trofinetide by Neuren’s global partner Acadia.

Neuren is looking forward to seeing the impact as DAYBUE STIX becomes more broadly available in 2026, he added.

Reason Behind the FDA Approval:

A Bioequivalence study which demonstrates the power formulation offering exposure than that of the original liquid version of DAYBUE, is a main support for the FDA decision to approving terofinetide’s new formulation to treat Rett syndrome. According to this study, patients who switch between formulation can gain the same efficacy and safety profile which established already in clinical trials.

Acadia’s Expectation from the Global Market:

Acadia is expecting DAYBUE STIX to be available in the first quarter of 2026, on a limited basis, and with great availability planning for early in the second quarter of 2026. The existing oral solution of trafinetide will also remain in the market.

Acadia holding exclusive global rights for developing and commercialize trofinetide, under its global licensing agreement, Neuren receiving royalties on all net sales, and the remaining for upcoming development and broad commercial payments.

About Trifonitide:

• Trofinotide is a synthetic analog of neurotrophic peptides, which are naturally occurring, and the first ever therapy being used to address the core symptoms of Rett syndrome.
• Rett syndrome is a neurodevelopmental disorder, emerges in early childhood and has historically lacked approved treatment options.
• This second FDA approval of the tridonitide formulation has expanded treatment options for children and adults who are suffering from Rett syndrome, providing families greater flexibility in dose management and modified tastes.